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Monoclonal Antibodies

ARGX-119 for Congenital Myasthenic Syndromes

Phase 1
Recruiting
Research Sponsored by argenx
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
At least 18 years of age.
Has genetically confirmed congenital myasthenic syndromes due to mutation of downstream of kinase 7 (DOK7-CMS).
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to week 42

Summary

This trial will test the safety and tolerability of a drug called ARGX-119 in adults with DOK7- Congenital Myasthenic Syndromes. It will also look at

Who is the study for?
This trial is for adults with a genetic condition called DOK7-Congenital Myasthenic Syndromes. Participants must be at least 18 years old and have been on a stable dose of oral beta agonists, like albuterol or ephedrine, for over three months.
What is being tested?
The study tests the safety and effects of ARGX-119 compared to a placebo in treating DOK7-CMS. Patients will receive intravenous infusions and be observed for approximately 11 months to see how their bodies react and if their symptoms improve.
What are the potential side effects?
While specific side effects are not listed, common ones may include reactions at the infusion site, potential immune system responses to ARGX-119, fatigue, headache, muscle or joint pain.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 18 years old or older.
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My condition is confirmed as DOK7-CMS through genetic testing.
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I have been on a stable dose of oral beta agonists for over 3 months and will not change it during the study.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to week 42
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to week 42 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Change from baseline over time for Myasthenia Gravis Activities of Daily Living (MG-ADL)
Change from baseline over time for Patient-Reported Outcomes Measurement Information System Global Health (PROMIS-GH) scale
Change from baseline over time for key components of the Quantitative Myasthenia Gravis (QMG) scale

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: ARGX-119Experimental Treatment1 Intervention
Participants receiving intravenous infusion of ARGX-119 once every other week
Group II: PlaceboPlacebo Group1 Intervention
Participants receiving intravenous infusion of placebo once every other week
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
ARGX-119
2023
Completed Phase 1
~120

Find a Location

Who is running the clinical trial?

argenxLead Sponsor
70 Previous Clinical Trials
11,049 Total Patients Enrolled
~10 spots leftby Oct 2025
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